Long-term treatment of SOD1 ALS with tofersen: a multicentre experience in 17 patients.

Mario SabatelliFederica CerriRiccardo ZuccarinoAgata Katia PatanellaDaniela BernardoGiulia BisogniRaffaella TanelValeria SansoneMassimiliano FilostoSerena LattanteFrancesco MartelloPaolo Niccolò DoronzioSalvatore StanoBruno Antonio ZanfiniMichela CocciaEmanuele Maria CostantiniAndrea LizioGabriele LucioliAlessandro PadovaniGian Paolo MerliniAmelia Conte

Published in: Journal of neurology (2024)

Clinical findings and NFL analysis strongly suggest that tofersen may have a disease-modifying effect in a subset of SOD1-ALS patients.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
amyotrophic lateral sclerosis
peritoneal dialysis
clinical trial
prognostic factors
randomized controlled trial
patient reported outcomes
study protocol
patient reported
replacement therapy